Cooperative Study of Factor VIII Inhibitors

NCT ID: NCT00000582

Last Updated: 2012-04-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Study Classification

INTERVENTIONAL

Study Start Date

1978-07-31

Study Completion Date

1979-07-31

Brief Summary

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To test the efficacy of prothrombin complex concentrates (Factor IX) in the treatment of hemophiliac patients who had inhibitors to Factor VIII.

Detailed Description

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BACKGROUND:

Despite major advances in the treatment of patients with hemophilia, a serious remaining challenge was presented by the occurrence of circulating inhibitors to Factor VIII. Because of lack of information on the natural course of patients with Factor VIII inhibitors, the relative efficacy of various modes of therapy was not established. The Division of Blood Diseases and Resources decided to sponsor a clinical investigation which would evaluate populations of hemophilia patients for Factor VIII inhibitors, follow up these patients to provide information on the natural history of the inhibitor in the hemophilia patients, and make available a reference center to monitor results and attain uniformity.

Treatment of a patient with a severe inhibitor and consequent bleeding remained a problem. Management included protracted treatment with Factor VIII, use of immunosuppressive agents and prothrombin complex (or Factor IX) concentrates. The rationale for Factor IX was that it bypassed the defect in Factor VIII caused by the inhibitor. This method of therapy attracted wide popularity, but the success was greatly debated. It was intended at the very outset of the Factor VIII study that therapeutic trials involving patients with inhibitors would not be a prime function, but that such studies would be monitored if necessary. A controlled trial of Factor IX concentrates therapy was strongly advised by the DBDR Advisory Committee. Accordingly, during fiscal year 1978, a protocol for a double-blind control study was developed by the Factor VIII inhibitor group. The trial began in the spring of 1978, and the intervention terminated about one year later.

DESIGN NARRATIVE:

Double-blind study; patients served as their own controls. A total of 51 patients each received a single large dose of Konyne, Proplex, or diluted albumin (as a control). Joint bleeding of the elbow, knee, and ankle was evaluated six hours after each dose.

Conditions

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Blood Coagulation Disorders Hematologic Diseases Hemophilia A Hemorrhagic Disorders

Study Design

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Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Interventions

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factor ix

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

Males with hemophilia, not stratified as to ethnic group.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role lead

Principal Investigators

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Louis Aledort

Role:

Icahn School of Medicine at Mount Sinai

J. Edson

Role:

University of Minnesota

M. Eyster

Role:

Milton S. Hershey Medical Center

Scott Goodnight

Role:

University of Oregon Health Sciences Center

William Hathaway

Role:

University of Colorado Medical Center

Jack Lazerson

Role:

Milwaukee Children's Hospital

Peter Levine

Role:

Memorial hospital

Jeanne Lusher

Role:

Wayne State University

Campbell McMillan

Role:

University of North Carolina

Sandor Shapiro

Role:

Thomas Jefferson University

References

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Eyster ME, Gill FM, Blatt PM, Hilgartner MW, Ballard JO, Kinney TR. Central nervous system bleeding in hemophiliacs. Blood. 1978 Jun;51(6):1179-88. No abstract available.

Reference Type BACKGROUND
PMID: 647123 (View on PubMed)

Lusher JM, McMillan CW. Severe factor VIII and factor IX deficiency in females. Am J Med. 1978 Oct;65(4):637-48. doi: 10.1016/0002-9343(78)90852-5.

Reference Type BACKGROUND
PMID: 309282 (View on PubMed)

Kasper CK. Effect of prothrombin complex concentrates on factor VIII inhibitor levels. Blood. 1979 Dec;54(6):1358-68.

Reference Type BACKGROUND
PMID: 508942 (View on PubMed)

Lusher JM, Shapiro SS, Palascak JE, Rao AV, Levine PH, Blatt PM. Efficacy of prothrombin-complex concentrates in hemophiliacs with antibodies to factor VIII: a multicenter therapeutic trial. N Engl J Med. 1980 Aug 21;303(8):421-5. doi: 10.1056/NEJM198008213030803.

Reference Type BACKGROUND
PMID: 6771653 (View on PubMed)

Gerety RJ, Eyster ME, Tabor E, Drucker JA, Lusch CJ, Prager D, Rice SA, Bowman HS. Hepatitis B virus, hepatitis A virus and persistently elevated aminotransferases in hemophiliacs. J Med Virol. 1980;6(2):111-8. doi: 10.1002/jmv.1890060203.

Reference Type BACKGROUND
PMID: 6264019 (View on PubMed)

Other Identifiers

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302

Identifier Type: -

Identifier Source: org_study_id

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